Stealth BioTherapeutics Announces SBT-272 Granted Orphan Drug Designation For Treatment Of ALS

SBT-272 Phase 1 safety and tolerability data support further clinical development

SBT-272 was neuroprotective and reduced neuroinflammation in ALS preclinical model

SBT-272 Granted Orphan Drug Designation for Treatment of ALS

BOSTON, Nov. 1, 2022 /PRNewswire/ -- Stealth BioTherapeutics Corp (NASDAQ:MITO), a clinical-stage biotechnology company focused on the discovery, development, and commercialization of novel therapies for diseases involving mitochondrial dysfunction, today announced encouraging Phase 1 data on the safety and tolerability of SBT-272, a next-generation small molecule designed to reach therapeutic concentrations in the brain and to restore mitochondrial structure and function.  The Company also announced that positive preclinical data demonstrating that treatment with SBT-272 provided neuroprotection of upper motor neurons harboring ALS pathology will be presented today at the Northeast Amyotrophic Lateral Sclerosis (NEALS) conference in Clearwater, FL. The Company recently received Orphan Drug Designation from the US Food and Drug Administration (FDA) Office of Orphan Products Development for SBT-272 for the treatment of patients with ALS.

"The preclinical data demonstrate that SBT-272 improves the stability and function of mitochondria in upper motor neurons that are diseased with TDP-43 pathology. This also provides neuroprotection and reduces neuroinflammation in the motor cortex of a TDP-43 model of ALS", said Hande Ozdinler, PhD, Associate Professor of Neurology, Feinberg School of Medicine, Northwestern University. "There appears to be compelling support for the therapeutic potential of targeting mitochondria in ALS and the ongoing clinical development of SBT-272."

Interim results from the Phase 1 study for SBT-272 demonstrated that selected doses are anticipated to result in therapeutic concentrations in the brain based on observed drug levels achieved in preclinical studies.  While final safety analyses are ongoing, dose levels identified for subsequent clinical evaluation appear generally safe and well tolerated. These data support further clinical development of SBT-272.

"We are pleased to receive orphan drug designation from FDA for SBT-272 for the treatment of ALS, underscoring the urgent need for innovative new therapies for this devastating disease," said Chief Executive Officer Reenie McCarthy. "We are encouraged by the clinical profile of SBT-272 observed to date and look forward to evaluating its therapeutic potential in ALS and other neurodegenerative diseases of mitochondrial dysfunction."

NEALS Conference November 1, 2022

Translational Pharmacology of SBT-272, A Novel Mitochondria-Targeted Drug for the Treatment of Neurodegenerative Disease

Poster presentation, November 1, from 5-7pm

SBT-272 improved mitochondria structure and function and preserved Upper Motor Neurons with TDP-43 Pathology

Platform presentation, November 3, 11:45 am